{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inherited+Metabolic+Storage+Diseases&page=10",
    "query": {
      "condition": "Inherited Metabolic Storage Diseases",
      "page": 10
    },
    "page_size": 10
  },
  "pagination": {
    "page": 10,
    "page_size": 10,
    "total_count": 595,
    "total_pages": 60,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inherited+Metabolic+Storage+Diseases&page=11&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inherited+Metabolic+Storage+Diseases&page=9&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-10T19:12:09.658Z",
  "attribution": "Data derived from public ClinicalTrials.gov records. The official record at https://clinicaltrials.gov/ remains the source of truth for current availability, contacts, and full study details.",
  "notes": [
    "This endpoint exposes only public summary fields — no participant contact emails, phone numbers, or scraped investigator contact details.",
    "For contact information and full protocol detail, follow each trial's `official_url` to the ClinicalTrials.gov record.",
    "This API is a navigational aid. It does not provide medical advice, eligibility determinations, or compensation guarantees."
  ],
  "trials": [
    {
      "nct_id": "NCT06910813",
      "title": "DFT383 in Pediatric Participants With Nephropathic Cystinosis",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Nephropathic Cystinosis"
      ],
      "interventions": [
        {
          "name": "DFT383",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "Novartis Pharmaceuticals",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "5 Years",
        "sex": "ALL",
        "summary": "2 Years to 5 Years"
      },
      "enrollment_count": 30,
      "start_date": "2025-06-02",
      "completion_date": "2044-05-28",
      "has_results": false,
      "last_update_posted_date": "2026-09-22",
      "last_synced_at": "2026-10-10T19:12:09.658Z",
      "location_count": 6,
      "location_summary": "Phoenix, Arizona • San Diego, California • Stanford, California + 3 more",
      "locations": [
        {
          "city": "Phoenix",
          "state": "Arizona"
        },
        {
          "city": "San Diego",
          "state": "California"
        },
        {
          "city": "Stanford",
          "state": "California"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06910813"
    },
    {
      "nct_id": "NCT07169279",
      "title": "Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Achondroplasia"
      ],
      "interventions": [
        {
          "name": "Infigratinib is provided as a single dose of minitablets for oral administration",
          "type": "DRUG"
        },
        {
          "name": "Infigratinib is provided as sprinkle capsules for daily oral administration",
          "type": "DRUG"
        },
        {
          "name": "Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "QED Therapeutics, a BridgeBio company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "0 Years",
        "maximum_age": "32 Months",
        "sex": "ALL",
        "summary": "0 Years to 32 Months"
      },
      "enrollment_count": 77,
      "start_date": "2025-11-19",
      "completion_date": "2032-03",
      "has_results": false,
      "last_update_posted_date": "2026-07-13",
      "last_synced_at": "2026-10-10T19:12:09.658Z",
      "location_count": 3,
      "location_summary": "Oakland, California • Baltimore, Maryland • Madison, Wisconsin",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Baltimore",
          "state": "Maryland"
        },
        {
          "city": "Madison",
          "state": "Wisconsin"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07169279"
    },
    {
      "nct_id": "NCT03612869",
      "title": "Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)",
      "overall_status": "UNKNOWN",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2",
        "PHASE3"
      ],
      "conditions": [
        "Mucopolysaccharidosis Type IIIA"
      ],
      "interventions": [
        {
          "name": "LYS-SAF302",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "LYSOGENE",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Months",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "6 Months and older"
      },
      "enrollment_count": 20,
      "start_date": "2018-12-17",
      "completion_date": "2022-03",
      "has_results": false,
      "last_update_posted_date": "2021-08-31",
      "last_synced_at": "2026-10-10T19:12:09.658Z",
      "location_count": 4,
      "location_summary": "Orange, California • Minneapolis, Minnesota • New York, New York + 1 more",
      "locations": [
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03612869"
    },
    {
      "nct_id": "NCT05699265",
      "title": "Cardiovasculorenal Phenotyping in Fabry Disease Through Noninvasive Testing",
      "overall_status": "TERMINATED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "Measures of arterial stiffness and endothelial function",
          "type": "DIAGNOSTIC_TEST"
        },
        {
          "name": "Ambulatory blood pressure monitoring",
          "type": "DIAGNOSTIC_TEST"
        },
        {
          "name": "Cardiopulmonary exercise testing (CPET)",
          "type": "DIAGNOSTIC_TEST"
        },
        {
          "name": "Serum and urine biomarkers",
          "type": "DIAGNOSTIC_TEST"
        }
      ],
      "intervention_types": [
        "DIAGNOSTIC_TEST"
      ],
      "sponsor": "Children's Hospital Medical Center, Cincinnati",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "8 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "8 Years and older"
      },
      "enrollment_count": 4,
      "start_date": "2023-02-01",
      "completion_date": "2025-04-01",
      "has_results": false,
      "last_update_posted_date": "2025-06-19",
      "last_synced_at": "2026-10-10T19:12:09.658Z",
      "location_count": 1,
      "location_summary": "Cincinnati, Ohio",
      "locations": [
        {
          "city": "Cincinnati",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05699265"
    },
    {
      "nct_id": "NCT00068107",
      "title": "Dosing Study of Replagal in Patients With Fabry Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "Replagal",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Baylor Research Institute",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "MALE",
        "summary": "Male only"
      },
      "enrollment_count": 13,
      "start_date": "2003-09",
      "completion_date": "2013-12",
      "has_results": true,
      "last_update_posted_date": "2015-03-31",
      "last_synced_at": "2026-10-10T19:12:09.658Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00068107"
    },
    {
      "nct_id": "NCT05845749",
      "title": "Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "MPS IVA",
        "MPS VI"
      ],
      "interventions": [
        {
          "name": "Vosoritide Injection [Voxzogo]",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "University of California, San Francisco",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "5 Years",
        "maximum_age": "10 Years",
        "sex": "ALL",
        "summary": "5 Years to 10 Years"
      },
      "enrollment_count": 6,
      "start_date": "2023-09-25",
      "completion_date": "2029-12-31",
      "has_results": false,
      "last_update_posted_date": "2026-03-10",
      "last_synced_at": "2026-10-10T19:12:09.658Z",
      "location_count": 1,
      "location_summary": "Oakland, California",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05845749"
    },
    {
      "nct_id": "NCT01387854",
      "title": "Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "MPS VI"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "BioMarin Pharmaceutical",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 59,
      "start_date": "2011-06",
      "completion_date": "2013-02",
      "has_results": false,
      "last_update_posted_date": "2014-04-02",
      "last_synced_at": "2026-10-10T19:12:09.658Z",
      "location_count": 1,
      "location_summary": "Oakland, California",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01387854"
    },
    {
      "nct_id": "NCT01869270",
      "title": "Gene Therapy for Tay-Sachs Disease",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Tay Sachs Disease",
        "Sandhoff Disease",
        "Late Onset Tay Sachs Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 4,
      "start_date": "2010-12",
      "completion_date": "2014-08",
      "has_results": false,
      "last_update_posted_date": "2014-12-04",
      "last_synced_at": "2026-10-10T19:12:09.658Z",
      "location_count": 2,
      "location_summary": "Tampa, Florida • Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Tampa",
          "state": "Florida"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01869270"
    },
    {
      "nct_id": "NCT00005111",
      "title": "PET Scans in Normal Volunteers and Patients With Fabry Disease",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Fabry Disease",
        "Healthy"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Institute of Neurological Disorders and Stroke (NINDS)",
      "sponsor_class": "NIH",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "MALE",
        "summary": "Male only"
      },
      "enrollment_count": 24,
      "start_date": "2000-04",
      "completion_date": "2001-03",
      "has_results": false,
      "last_update_posted_date": "2008-03-04",
      "last_synced_at": "2026-10-10T19:12:09.658Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00005111"
    },
    {
      "nct_id": "NCT01275066",
      "title": "A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "MPS IV A"
      ],
      "interventions": [
        {
          "name": "BMN 110 Weekly",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        },
        {
          "name": "BMN 110 Every Other Week",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "BioMarin Pharmaceutical",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "5 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "5 Years and older"
      },
      "enrollment_count": 177,
      "start_date": "2011-02",
      "completion_date": "2012-08",
      "has_results": true,
      "last_update_posted_date": "2014-07-07",
      "last_synced_at": "2026-10-10T19:12:09.658Z",
      "location_count": 6,
      "location_summary": "Oakland, California • Wilmington, Delaware • Washington D.C., District of Columbia + 3 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Wilmington",
          "state": "Delaware"
        },
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01275066"
    }
  ]
}